Featured Research

New gene therapy proves effective in treating severe heart failure

Date:

June 5, 2010

Source:

The Mount Sinai Hospital / Mount Sinai School of Medicine

Summary:

Researchers have developed a new gene therapy that is safe and effective in reversing advanced heart failure. SERCA2a is a gene therapy designed to stimulate production of an enzyme that enables the failing heart to pump more effectively. In a Phase II study, SERCA2a injection through a routine minimally invasive cardiac catheterization was safe and showed clinical benefit in treating this patient population and decreasing the severity of heart failure.

Share This

Researchers at Mount Sinai School of Medicine have developed a new gene therapy that is safe and effective in reversing advanced heart failure. SERCA2a (produced as MYDICARฎ) is a gene therapy designed to stimulate production of an enzyme that enables the failing heart to pump more effectively. In a Phase II study, SERCA2a injection through a routine minimally invasive cardiac catheterization was safe and showed clinical benefit in treating this patient population and decreasing the severity of heart failure.

Related Articles

The data were presented recently at the Heart Failure Congress of the European Society of Cardiology in Berlin.

"SERCA2a met the primary endpoints and appears to be safe and effective in people with advanced heart failure," said trial investigator Jill Kalman, MD, Associate Professor, Medicine, Cardiology, Director of the Cardiomyopathy Program, Mount Sinai School of Medicine. "There is a significant unmet need for treatments in this patient population, and these data indicate that SERCA2a is a promising option for them."

The CUPID (Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease) trial is a randomized, double-blind, placebo-controlled study, which enrolled 39 patients with advanced heart failure to study the safety and efficacy of SERCA2a. Patients were randomized to receive SERCA2a gene delivery in one of three doses or placebo and were evaluated over six months. The treatment is delivered directly to the patient's heart during a routine outpatient catheterization procedure.

Patients in the SERCA2a group demonstrated improvement or stabilization in symptoms, heart function, and severity of heart failure. They also saw an increase in time between cardiovascular events and a decrease in frequency of events. SERCA2a was found to be safe, with no increases in adverse events, disease-related events, laboratory abnormalities, or arrhythmias compared to placebo.

SERCA2a was developed by a team led by Roger J. Hajjar, MD, Research Director of Mount Sinai's Wiener Family Cardiovascular Research Laboratories and the Arthur & Janet Ross Professor of Cardiology, Medicine, and Gene and Cell Medicine, Mount Sinai School of Medicine. The team discovered the landmark potential of the treatment in 1999 and has been pursuing its potential as a gene therapy target in state-of-the-art specially built pre-clinical laboratories at Mount Sinai.

"Mount Sinai Heart is committed to developing ground-breaking therapies and bringing them from bench to bedside," said Valentin Fuster, MD, Director of Mount Sinai Heart, the Zena and Michael A. Wiener Cardiovascular Institute and the Marie-Josee and Henry R. Kravis Center for Cardiovascular Health, The Mount Sinai Medical Center. "We look forward to further study of this important treatment."

According to the U.S. Centers for Disease Control & Prevention, about 5.8 million Americans suffer from heart failure, and 670,000 new cases are diagnosed each year. One in five people who have heart failure die within one year of diagnosis. In 2010, heart failure will cost the United States $39.2 billion, including the cost of health care services, medications, and lost productivity. Heart failure is most often treated with aggressive medical and device therapy, but has no cure. The most common symptoms of heart failure are shortness of breath, feeling tired, and swelling in the ankles, feet, legs, and sometimes the abdomen.

The CUPID Trial is funded by Celladon Corporation, makers of MYDICAR. The company was co-founded by Dr. Hajjar who has an equity interest in Celladon Corporation and participates on an Advisory Board.

Mar. 31, 2015  Increasing state alcohol taxes could prevent thousands of deaths a year from car crashes, say researchers, who found alcohol-related motor vehicle crashes decreased after taxes on beer, wine and ... full story

Mar. 31, 2015  Alcoholism takes a toll on every aspect of a person's life, including skin problems. Now, a new research report helps explain why this happens and what might be done to address it. "The clinical ... full story

Mar. 31, 2015  A new population of 'memory' immune cells has been discovered by scientists, throwing light on what the body does when it sees a microbe for the second time. This insight, and others like it, will ... full story

Mar. 31, 2015  Coronary heart disease and stroke, two of the leading causes of death in the United States, are diseases associated with heightened platelet reactivity. A new study in humans suggests an underlying ... full story

Mar. 31, 2015  A new study had researchers seeking answers to why the therapeutic benefit afforded by SSRIs was so limited in children and teenagers. If researchers can uncover the biological mechanisms preventing ... full story

Mar. 31, 2015  A drug being developed to treat osteoporosis may also be useful for treating osteogenesis imperfecta or brittle bone disease, a rare but potentially debilitating bone disorder that that is present ... full story

Mar. 31, 2015  It is possible to quantify and classify the effects of different diseases on the activity of intestinal bacteria, new research demonstrates for the first time. Human intestinal flora, known as ... full story

Mar. 31, 2015  During prenatal development, the brains of most animals, including humans, develop specifically male or female characteristics. But scientists have known little about the details of how this ... full story

Mar. 31, 2015  A history of depression may put women at risk for developing diabetes during pregnancy, according to research. This study also pointed to how common depression is during pregnancy and the need for ... full story

Featured Videos

Solitair Device Aims to Takes Guesswork out of Sun Safety

Reuters - Innovations Video Online (Mar. 31, 2015)  The Solitair device aims to take the confusion out of how much sunlight we should expose our skin to. Small enough to be worn as a tie or hair clip, it monitors the user&apos;s sun exposure by taking into account their skin pigment, location and schedule. Matthew Stock reports.
Video provided by Reuters

Soda, Salt and Sugar: The Next Generation of Taxes

Washington Post (Mar. 30, 2015)  Denisa Livingston, a health advocate for the Din้ Community Advocacy Alliance, and the Post&apos;s Abby Phillip discuss efforts around the country to make unhealthy food choices hurt your wallet as much as your waistline.
Video provided by Washington Post

S. Leone in New Anti-Ebola Lockdown

AFP (Mar. 28, 2015)  Sierra Leone imposed a three-day nationwide lockdown Friday for the second time in six months in a bid to prevent a resurgence of the deadly Ebola virus. Duration: 01:17
Video provided by AFP

Sep. 7, 2011  Researchers have identified a new drug target that may treat and/or prevent heart failure. The team evaluated failing human and pig hearts and discovered that SUMO1, a so-called "chaperone" ... full story

June 28, 2011  Researchers have found in a Phase II trial that a new gene therapy stabilized or improved cardiac function in people with severe heart failure. Patients receiving a high dose of the therapy, called ... full story

ScienceDaily features breaking news and videos about the latest discoveries in health, technology, the environment, and more -- from major news services and leading universities, scientific journals, and research organizations.